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Gene correction of HBB mutations in CD34+ hematopoietic stem cells using Cas9 mRNA and ssODN donors

14 Nov 2018

Abstract excerpt

β-Thalassemia is an inherited hematological disorder caused by mutations in the human hemoglobin beta ( HBB ) gene that reduce or abrogate β-globin expression. Although lentiviral-mediated expression of β-globin and autologous transplantation is a promising therapeutic approach, the risk of insertional mutagenesis or low transgene expression is apparent. However, targeted gene correction of HBB mutations with...

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PMID: 30430274
DOI: 10.1186/s40348-018-0086-1

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